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Onasemnogene abeparvovec-xioi (Zolgensma): Approval strategy with manipulated data?

August 13, 2019 – Onasemnogene abeparvovec-xioi (Zolgensma) is an adeno-associated virus vector-based gene therapy indicated for the treatment of pediatric patients less than 2 years of age with spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene.

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Gene therapy: Zolgensma to treat spinal muscular atrophy

May 29, 2019 – Spinal muscular atrophy (SMA) is a group of neuromuscular disorders that result in the loss of motor neurons and progressive muscle wasting. The severity of symptoms and age of onset varies by the type. Some types are apparent at or before birth while others are not apparent until adulthood. 

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  • Ancestry launches DNA health service that will compete with 23andMe October 15, 2019
    Ancestry has long appealed to people eager to learn about their family roots. Now the company known for its popular DNA kits is diving into a new area of discovery—health.
  • Improving research with more effective antibodies October 15, 2019
    A new study points to the need for better antibody validation, and outlines a process that other labs can use to make sure the antibodies they work with function properly.
  • New DNA 'clock' could help measure development in young children October 15, 2019
    Scientists have developed a molecular "clock" that could reshape how pediatricians measure and monitor childhood growth and potentially allow for an earlier diagnosis of life-altering development disorders.
  • DNA fracturing rewires gene control in cancer October 15, 2019
    Understanding the mechanisms that mediate widespread DNA damage in the cancer genome is of great interest to cancer physicians and scientists because it may lead to improved treatments and diagnosis. In this study, a multi-institutional team led by researchers at Baylor College of Medicine has brought attention to genomic structural variation as a previously unappreciated […]
  • Most genetic studies use only white participants – this will lead to greater health inequality October 15, 2019
    Few areas of science have seen such a dramatic development in the last decade as genomics. It is now possible to read the genomes of millions of people in so-called genome-wide association studies. These studies have identified thousands of small differences in our genome that are linked to diseases, such as cancer, heart disease and […]
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